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Sangamo Therapeutics, Inc. (previously known as Sangamo Biosciences, Inc.) is an American biotechnology company based in Brisbane, California. It applies cell and gene therapy to combat haemophilia and other genetic diseases.
Sangamo Therapeutics, Inc. (previously known as Sangamo Biosciences, Inc.) is an American biotechnology company based in Brisbane, California. It applies cell and gene therapy to combat haemophilia and other genetic diseases.
History
The company was founded in 1995 in Richmond, California. It was originally known as Sangamo Biosciences, Inc. before changing names in 2017. In September 2018, it had 182 employees. Sandy Macrae is the president. In 2018,Edward Rebar
Edward John Rebar is an American biologist, and is Senior Vice President and Chief Technology Officer of Sana Biotechnology.
Education
Edward John Rebar earned a Bachelor of Science in biochemistry from Rutgers University. He completed a Doct ...
became the senior vice president and chief technology officer of Sangamo.
Research
Sangamo applies technology to treat haemophilia B and lysosomal storage diseases includingmucopolysaccharidosis type I
Mucopolysaccharidosis type I is a spectrum of diseases in the mucopolysaccharidosis family. It results in the buildup of glycosaminoglycans (or GAGs, or mucopolysaccharides) due to a deficiency of alpha-L iduronidase, an enzyme responsible for t ...
(Hurler Syndrome) and mucopolysaccharidosis type II (Hunter Syndrome). The FDA granted Sangamo fast track designation for SB-525, a gene therapy candidate for haemophilia A. In its partnership with Pfizer in 2017, Sangamo uses Bioverativ
Bioverativ Inc. is an American multinational biotechnology company that specializes in the discovery, development, and delivery of therapies for the treatment of haemophilia. Bioverativ competes with Baxalta (acquired by Shire Plc in 2016), Pfi ...
in hemoglobinopathies
Hemoglobinopathy is the medical term for a group of inherited blood disorders and diseases that primarily affect red blood cells. They are single-gene disorders and, in most cases, they are inherited as autosomal co-dominant traits.
There are t ...
such as beta thalassemia and sickle cell disease. It is also developing zinc finger gene editing technology.
In February 2019, medical scientists, working with Sangamo Therapeutics, announced the first ever "in body" human gene editing therapy
Genome editing, or genome engineering, or gene editing, is a type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism. Unlike early genetic engineering techniques that randomly inserts g ...
to permanently alter DNA - in a patient with Hunter syndrome. clinical trials by Sangamo involving gene editing using zinc finger nuclease were ongoing.
Clinical trials
Sangamo's programs are a mix of wholly owned and partnered; major partners include Pfizer, Biogen, Sanofi, Takeda, and Kite (a Gilead company). As of 2020 Sangamo had the following clinical trials underway in the US: * Phase 1/2 Thales study in beta thalassemia * Phase 1/2 STAAR study in Fabry disease * Phase 1/2 PRECIZN-1 study in sickle cell disease * Phase 1/2 CHAMPIONS study inMPS II
Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. It is a form of lysosomal storage disease. ...
* Phase 1/2 EMPOWERS study in MPS I
* Phase 1/2 Alta study in hemophilia A
* Phase 1 FIXTENDZ study in hemophilia B
In February 2023, Sangamo Therapeutics announced that it would stop the late-stage development for the Phase 1/2 PRECIZN-1 study of BIVV003 its sickle cell drug.
See also
*Alan Wolffe
Alan Wolffe (21 June 1959 – 26 May 2001) was an English cell biologist known for his prominent role in establishing that the chromosomal organisation of genes is a dynamic phenomenon determining their expression, cell division and differentia ...
References
External links
* {{DEFAULTSORT:Sangamo Therapeutics Companies based in San Mateo County, California Biotechnology companies of the United States 1995 establishments in California Orphan drug companies Biotechnology companies established in 1995 Health care companies based in California